Deal calls for $217 million in development, regulatory, and commercial milestones for the new program.
Acceleron Pharma and Celgene entered into a joint development and commercialization agreement for ACE-536 for the treatment of anemia, with Celgene paying $25 million up front. Acceleron is eligible to receive development, regulatory, and commercial milestones of up to $217 million for the ACE-536 program.
Additionally, Celgene will have an option to future Acceleron candidates developed for anemia. The companies already have a collaboration around ACE-011 (sotatercept), which was inked in 2008. “Acceleron has uncovered an exciting new approach to treating disorders of erythropoiesis,” says John Knopf, Ph.D., CEO of Acceleron. “We look forward to initiating the Phase I clinical trial of ACE-536 within the next few months.”
Under terms of the agreement, Acceleron will be responsible for conducting the Phase I and initial Phase II trials as well as manufacturing ACE-536 for these studies. Celgene will conduct subsequent Phase II and Phase III trials and also take over production work.
Acceleron will pay a share of the development expenses through the end of 2012, and Celgene will be responsible for development costs thereafter. The companies will co-promote the products in North America. Acceleron will receive tiered double-digit royalties on worldwide net sales.
ACE-536 is a ligand trap that inhibits members of the TGF-beta superfamily involved in late stages of erythropoiesis. ACE-536 and sotatercept are biochemically distinct molecules and may have unique pharmacological attributes that enable their preferential use in particular anemia indications, the companies report.
In preclinical studies ACE-536 promoted red blood cell (RBC) formation in the absence of erythropoietin (EPO) signaling, had distinct effects from EPO on RBC differentiation, and acted on a different population of progenitor blood cells than EPO during RBC development.
ACE-011 is a soluble receptor fusion protein comprised of extracellular domain of the human activin receptor type IIA (ActRIIA) fused to human immunoglobulin. Blocking signaling through ActRIIA may be a way to increase red blood cell production, promote bone formation, and inhibit tumor growth and metastasis, Acceleron explains. The drug is currently being studied in Phase II trials in patients with chemotherapy-induced anemia and in patients with end-stage renal disease on hemodialysis.